On May 11, 2021 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious blood diseases, reported financial results for the quarter ended March 31, 2021 (Press release, Gamida Cell, MAY 11, 2021, View Source [SID1234579705]). The company also highlighted progress with omidubicel, an advanced cell therapy with positive Phase 3 clinical data, as a potentially life-saving treatment option for patients in need of an allogeneic hematopoietic stem cell (bone marrow) transplant, and GDA-201, a natural killer (NK) cell immunotherapy in Phase 1/2 development for patients with non-Hodgkin lymphoma (NHL)
"In the first quarter of this year, we made significant progress on key initiatives across all functions of our business, starting with omidubicel, a potentially transformative treatment option for patients with hematological malignancies," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We are working diligently to bring this novel therapy to patients, with submission of a BLA to the FDA anticipated in the fourth quarter of this year. We are progressing well with our manufacturing readiness activities in response to the clear feedback from the FDA regarding registration of our commercial manufacturing facilities and are actively building our launch readiness capabilities, including market access and support services, to ensure a positive patient experience at transplant at the time of potential FDA approval."

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We also continue to expand our clinical pipeline with plans to submit an IND for our GDA-201 natural killer cell therapy, initiate a multi-center Phase 1/2 clinical study in NHL and continue to advance our R&D activities to pursue the development of genetically modified NAM-enabled NK cells in solid tumors. Importantly, we are well positioned to deliver our 2021 corporate goals and objectives toward improving the lives of the patients we serve," Dr. Adams continued.

Omidubicel, a proprietary, investigational advanced cell therapy for allogeneic bone marrow transplant

Omidubicel is the foundational product based on Gamida Cell’s proprietary cell expansion technology. During the quarter, Gamida Cell continued to advance omidubicel, the first cell therapy for bone marrow transplant to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA). The company anticipates submitting a Biologics License Application (BLA) to the FDA in the fourth quarter of this year, based on the results of an international, randomized Phase 3 study of omidubicel that was designed to evaluate the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing a bone marrow transplant compared to patients who received a standard umbilical cord blood transplant. The study achieved its primary endpoint, a statistically significant reduction in time to neutrophil engraftment, as well as all key secondary endpoints. A key milestone in a patient’s recovery, neutrophil engraftment is a measure of how quickly the stem cells a patient receives in a bone marrow transplant are established and begin to make healthy new cells. In the recently completed Phase 3 study, the median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p < 0.001). Additionally, the study met key secondary endpoints related to the speed of platelet engraftment, decrease in infections and reduction in hospitalizations, all significant clinical measures in bone marrow transplant.

In February 2021, the company presented details of the results of the omidubicel Phase 3 study at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research. The study’s intent-to-treat analysis included 125 patients aged 13–65 years with a median age of 41. Patients were enrolled at more than 30 clinical centers in the United States, Europe, Asia, and Latin America. Racial and ethnic diversity and baseline characteristics which were well-balanced across the two study groups. Diseases included acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma.

In addition to the efficacy results described above, safety results were also presented, showing decreased incidence related to grade III/IV acute GvHD (14 percent for omidubicel, 21 percent for the comparator) and comparable results for all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Transplants with umbilical cord blood, the comparator, have been historically shown to result in low incidence of GvHD in relation to other graft sources and, in this study, omidubicel demonstrated a similar GvHD profile.

The data from the study relating to exploratory endpoints also supported the clinical benefit demonstrated by the study’s primary and secondary endpoints. The rate of infection was significantly reduced for patients randomized to omidubicel, with the cumulative incidence of first grade II or grade III bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.027). Additionally, the study demonstrated a reduction in the incidence of viral infections. Non-relapse mortality was 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09). Overall survival at 15 months following randomization was 73 percent for patients randomized to omidubicel and 62 percent for patients randomized to control (p=0.16), median overall survival was not yet reached. Non-relapse mortality and overall survival were exploratory endpoints that were not powered for statistical significance. When considering the patient experience following transplant, faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital are all meaningful results and represent potentially important advancements in care. Learn more.

Gamida Cell also reported data from the Phase 3 study in March 2021, in an oral session at the Presidential Symposium of the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2021). Additionally, the session was featured in a panel discussion, "EBMT Talks: Live with the Best Abstracts."

Additional omidubicel highlights:

Progress with commercial manufacturing readiness: Gamida Cell is making important progress to address the clear feedback received during a Type B meeting with the FDA in December 2020 for commercial manufacturing facilities to be ready for BLA submission. These facilities include the Gamida Cell facility in Israel and a commercial facility for which the company has a contractual relationship with Lonza. Both of these facilities are currently on track to meet the FDA requirements that will be required for BLA submission.
Continued launch readiness: The company continues to progress commercial launch readiness activities for the potential launch of omidubicel in 2022, pending FDA approval. Based on market research insights, there is a clear opportunity to improve outcomes based on clinical needs with current donor sources, increase access for patients who are eligible and not matched for transplant, and increase patient eligibility based on the encouraging clinical profile of omidubicel.
Gamida Cell announced the Gamida Cell Assist program. The transplant process can be challenging and complex for patients, caregivers and the entire transplant care team. Gamida Cell Assist is designed to focus on patient access and support at each step of the process. Once the program is launched, the Gamida Cell Assist case management team will provide a consistent, single point of contact for patients and health care professionals, work with the transplant center to track production of omidubicel for each individual patient, and provide real-time updates on the status of the therapy. The services provided will include coverage and reimbursement support, which may include financial, travel and lodging assistance. Gamida Cell is committed to supporting a positive journey for patients and their transplant teams so they can focus on what matters most, the patient experience and successful clinical outcomes. Learn more.
Phase 1/2 study of omidubicel in patients with severe aplastic anemia: Gamida Cell is actively evaluating omidubicel in an investigator-sponsored Phase 1/2 study in patients with severe aplastic anemia (SAA). Results to date have shown that omidubicel can result in rapid engraftment and can achieve sustained hematopoiesis in patients who are at high risk for graft failure with conventional umbilical cord blood transplant.
GDA-201, a proprietary innate NK cell immunotherapy

Continued advancement of Phase 1/2 study of GDA-201: Gamida Cell is preparing for the submission of an investigational new drug (IND) application for cryopreserved, off-the-shelf GDA-201 to enable a multi-center, Phase 1/2 clinical study in patients with NHL in the second half of this year. Gamida Cell is pioneering a potentially curative, novel approach that harnesses the power of its cell expansion technology, which improves antibody-dependent cellular cytotoxicity and tumor targeting of NK cells. Learn more.
Advancing NK cell R&D activities: The company continues to advance R&D activities to support pipeline growth, including the development of genetically modified NK cells.
Corporate Highlights

Strengthened financial position: In February 2021, the company completed a $75 million financing with Highbridge Capital Management, LLC, before deducting offering expenses. This financing will be used to support manufacturing, regulatory and potential commercial development activities for omidubicel and to further the preclinical and clinical development of GDA-201.
First Quarter 2021 Financial Results

Research and development expenses in the first quarter of 2021 were $11.4 million, compared to $7.9 million for the same period in 2020. The increase was mainly due to omidubicel commercial manufacturing readiness activities and advancing the GDA-201 program, including broadening the company’s scientific capabilities and talent.
Commercial expenses in the first quarter of 2021 were $4.4 million compared to $1.5 million for the first quarter of 2020. The increase was mainly attributed to progress with commercial readiness activities, including the hiring of an experienced commercial leadership team.
General and administrative expenses were $3.4 million for the first quarter of 2021 compared to $3.0 million for the same period in 2020. The increase was mainly due to the hiring of key management positions to support the growth of the business.
Finance income, net, was $0.7 million for the first quarter of 2021, compared to finance income, net, of $1.7 million for the first quarter of 2020. The decrease was primarily due to interest expenses following the recent $75M financing with Highbridge Capital Management, and non-cash expense resulting from revaluation of warrants, and Israeli Innovation Authority royalty-bearing grant liability.
Net loss for the first quarter of 2021 was $18.0 million, compared to a net loss of $10.6 million for the same period in 2020.
As of March 31, 2021, Gamida Cell had total cash and cash equivalents of $174.8 million, compared to $127.2 million as of December 31, 2020.
2021 Financial Guidance

Gamida Cell expects cash used for ongoing operating activities in 2021 to range from $110 million to $120 million.

Gamida Cell expects that its current cash and cash equivalents will support the company’s ongoing operating activities into the second half of 2022. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding and any business development activities that may be undertaken.

Expected 2021-2022 Milestones and Key Events

Gamida Cell expects the following milestones and key events through 2022:

Omidubicel

BLA submission to the FDA in the fourth quarter of 2021
Manufacturing and launch readiness activities ongoing for potential FDA approval in 2022
GDA-201

Submit company-sponsored IND application to the FDA and initiate a Phase 1/2 clinical study in NHL patients in the second half of 2021
Conference Call Information

Gamida Cell will host a conference call today, May 11, 2021, at 8:00 a.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 5258448. A recording of the webcast will be available approximately two hours after the event, for approximately 30 days.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201, an innate NK cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.3 For more information on the clinical study of GDA-201, please visit www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

On May 11, 2021 Day One Biopharmaceuticals, a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for patients of all ages with genetically defined cancers, reported that the first patient has been dosed in FIREFLY-1, a Phase 2 clinical trial evaluating the safety and efficacy of DAY101 in pediatric, adolescent and young adult patients with recurrent or progressive low-grade glioma harboring a known BRAF alteration (Press release, Day One, MAY 11, 2021, View Source [SID1234579704]). Pediatric low-grade glioma (pLGG) is the most common form of childhood brain cancer and has no approved targeted therapeutics. The trial is being conducted in collaboration with the Pacific Pediatric Neuro-Oncology Consortium (PNOC) and is designed to support the regulatory approval of DAY101.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

DAY101 is an oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway. Dysregulation of the MAPK pathway has been shown to occur in many cancers. In pLGG, BRAF wild-type fusions are the most common cancer-causing genomic alterations. DAY101 has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration who require systematic therapy and who have either progressed following prior treatment or who have no satisfactory alternative treatment options.

"Treating the first patient in FIREFLY-1 is an important milestone for Day One and underscores our critical mission of advancing innovative targeted therapies for people of all ages living with cancer," said Jeremy Bender, Ph.D., chief executive officer of Day One. "Children with low-grade glioma often face surgery and years of increasingly aggressive therapies that can have lasting effects on learning, cognition, and quality of life. We look forward to working closely with PNOC to complete this trial, which has the potential to make DAY101 the first approved therapy for pLGG and the first approved pan-RAF inhibitor."

"Studies have demonstrated that DAY101 has high brain distribution and exposure, thus potentially benefiting patients with brain tumors such as pLGG," said Samuel Blackman, M.D., Ph.D., co-founder and chief medical officer of Day One. "The FIREFLY-1 pivotal trial follows initial data from the Phase 1 PNOC014 study in nine children with pLGG, which indicated that DAY101, as monotherapy, has potent anti-tumor activity, rapid onset of responses, and was well tolerated. We believe DAY101 has the potential to become an important treatment advance for pediatric patients with RAF-altered low-grade gliomas."

About FIREFLY-1

FIREFLY-1 is a pivotal Phase 2, multicenter, open-label study designed to evaluate the safety and efficacy of DAY101 in patients aged 6 months to 25 years with relapsed or progressive low-grade glioma harboring a known activating BRAF alteration. The study aims to enroll approximately 60 patients, who will receive oral DAY101 at a dose of 420 mg/m2 once weekly. The primary endpoint will be overall response rate (ORR), defined as the proportion of patients with best overall confirmed response rate based upon Response Assessment for Neuro-Oncology (RANO) criteria. Secondary and exploratory endpoints include the overall response rate based on Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria and volumetric analyses, event free survival, safety, functional outcomes, and quality of life measures.

Additional information about FIREFLY-1 may be found at ClinicalTrials.gov, using Identifier NCT04775485.

About Pediatric Low-Grade Glioma

Pediatric low-grade glioma (pLGG) is the most common brain tumor diagnosed in children, accounting for 30% – 50% of all central nervous system tumors. BRAF wild-type fusions are the most common cancer-causing genomic mutations in pediatric low-grade gliomas. These genomic alterations are also found in several adult solid tumors. Currently approved BRAF inhibitors are only active in tumors harboring BRAF V600 mutations, exhibit limited activity in brain tumors, and cannot be used in patients harboring BRAF fusions.

Pediatric low-grade glioma can impact a child’s health in many ways depending on tumor size and location, including vision loss and motor dysfunction. There are no approved therapies for pLGG and current treatment approaches are associated with significant acute and life-long adverse effects. While most children with pLGG survive their cancer, children who do not achieve a cure following surgery face years of increasingly aggressive therapies that can have lasting effects on learning, cognition, and quality of life. Due to the indolent nature of pLGG, patients receive multiple years of systemic therapy.

About DAY101

DAY101 is an oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway. Studies have shown DAY101 has high brain distribution and exposure in comparison to other MAPK pathway inhibitors, thus potentially benefiting patients with primary brain tumors or brain metastases of solid tumors. DAY101 is a type II RAF inhibitor that selectively inhibits both monomeric and dimeric RAF kinase, which broadens its potential clinical application to treat an array of RAF-altered tumors.

DAY101 has been studied in over 250 patients, and as a monotherapy demonstrated good tolerability and encouraging anti-tumor activity in pediatric and adult populations with specific MAPK pathway-alterations.

In November 2020, Day One announced preliminary results from PNOC014, an ongoing Phase 1 Pacific Pediatric Neuro-Oncology Consortium (PNOC) network study with DAY101 sponsored by the Dana-Farber Cancer Institute, in patients under 18 years of age with relapsed low-grade glioma. Preliminary results demonstrated that of the eight patients in the study with RAF fusions, two patients achieved a complete response by Response Assessment for Neuro-Oncology (RANO), three had a partial response, two achieved prolonged stable disease, and one experienced progressive disease. DAY101 also demonstrated a tolerable safety profile.

DAY101 has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration who require systemic therapy and who have either progressed following prior treatment or who have no satisfactory alternative treatment options. In addition, DAY101 has received Orphan Drug designation from the FDA for the treatment of malignant glioma.

Day One is conducting a pivotal Phase 2 trial (FIREFLY-1) of DAY101 in pediatric, adolescent and young adult patients with pLGG. Day One also plans to study DAY101 alone or in combination with other agents that target key signaling nodes in the MAPK pathway, such as the Company’s MEK inhibitor pimasertib, in patient populations where various RAS and RAF alterations are believed to play an important role in driving disease.

About the Pacific Pediatric Neuro-Oncology Consortium

The Pacific Pediatric Neuro-Oncology Consortium (PNOC) is an international consortium with study sites within the United States, Canada, Israel, Europe, and Australia dedicated to bringing new therapies to children and young adults with brain tumors.

On March 11, 2021 Catamaran Bio, Inc., a biotechnology company developing allogeneic CAR-NK cell therapies to treat solid tumors, reported that an oral presentation with results generated using an advanced transposon technology for non-viral engineering of CAR-NK cells will be highlighted at the virtual annual meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), based on work from the laboratory of the company’s scientific founder, Branden Moriarity, PhD (Press release, Catamaran Bio, MAY 11, 2021, View Source [SID1234579703]). This is the first presentation of data for this novel transposon technology in NK cells, demonstrating high efficiency transposon integration in primary NK cells, subsequent cell expansion, and in vitro activity of the resulting CAR-NK cells. Dr. Moriarity, an Assistant Professor at the Medical School at the University of Minnesota in the Division of Pediatric Hematology/Oncology, is a pioneer in the fields of genome engineering and non-viral cell engineering and a scientific co-founder of Catamaran. In addition to an ongoing research collaboration with Dr. Moriarity, Catamaran also received support through an early investment from the University of Minnesota Discovery Capital investment program.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We congratulate our collaborators at the University of Minnesota for this landmark presentation, which provides the first demonstration that this transposon technology efficiently generates active CAR-NK cells and offers potential advantages compared to viral vectors in the manufacture of CAR-NK cells," said Vipin Suri, PhD, Chief Scientific Officer of Catamaran Bio. "This novel transposon technology demonstrates important capabilities for improved manufacturability, which will be essential in developing safe and effective cell therapies for solid tumors. We look forward to working with Dr. Moriarity as we develop and advance our CAR-NK programs."

The oral presentation, entitled "Non-Viral Engineering of CAR-NK Cells Using the TC Buster Transposon System," will occur on Thursday, May 13, 2021, at 6:30 p.m. ET during the Advances in Cellular and Immunotherapies session of the ASGCT (Free ASGCT Whitepaper) virtual meeting. The data presented highlights the following research findings:

Approximately 50% transposon integration efficiency using the transposon technology to deliver a multi‑cistronic chimeric antigen receptor (CAR) cassette into primary human peripheral blood NK cells.
Resulting cells underwent greater than 800-fold expansion and demonstrated efficacy in an in vitro cell-killing assay.
In contrast to the viral gene delivery methods commonly used in cell therapy manufacturing today, DNA transposon technology has the capacity to efficiently deliver large multi‑cistronic DNA cargoes and drive multiplex genome engineering via a single electroporation step. Transposons have the potential for more convenient and cost-effective manufacture of cell therapy products.

"We are pleased to present these data showing the effective transposon integration in primary NK cells with our transposon technology, the rapid expansion of the resulting CAR-NK cells and their activity in an in vitro model," said Dr. Moriarity. "I look forward to continuing to work closely with the team at Catamaran to utilize this technology in conjunction with their CAR-NK cell therapy platform as they advance new cell therapy approaches to treat solid tumors."

About the TAILWIND Platform

Catamaran’s TAILWIND Platform integrates proprietary capabilities to create novel, allogeneic CAR‑NK cell therapies by harnessing the natural cancer-fighting properties of natural killer (NK) cells and enhancing them with the power of synthetic biology and innovative NK cell engineering and manufacturing. With the TAILWIND Platform, CAR-NK cells are programmed with NK cell-specific CAR architectures and potency-boosting switches to neutralize the hostile tumor microenvironment and enable efficacy against diverse cancer types, especially solid tumors. Additionally, the TAILWIND Platform includes proprietary, non-viral NK cell engineering technology for efficient modification of NK cells with customized genetic programs enabled by synthetic biology. Catamaran’s CAR-NK cell therapies use healthy donor cells that are engineered and manufactured for off‑the‑shelf use, unlike current CAR-T cell therapies that use a patient’s own genetically modified T cells and require a customized, multi-week manufacturing process.

On May 11, 2021 Takeda Pharmaceutical Company Limited (TOKYO:4502) (NYSE:TAK) ("Takeda") reported financial results for fiscal year 2020 (period ended March 31, 2021) (Press release, Takeda, MAY 11, 2021, View Source [SID1234579702]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

TAKEDA PRESIDENT AND CHIEF EXECUTIVE OFFICER CHRISTOPHE WEBER commented:

"Over the course of FY2020, Takeda remained resilient as we operated in new ways through the COVID-19 pandemic. This is a testament to the dedication of our employees, and Takeda’s unwavering commitment to serving patients, our people, and the planet to bring better health for people and a brighter future for the world. We maintained business continuity, ensured patient access to our medicines and safeguarded the health and well-being of our employees while helping to address the pandemic.

"This focus enabled us to deliver on our full-year management guidance, with underlying revenue growth driven by our 14 global brands, and the acceleration of cost synergies contributed to strong margins and a robust cashflow. Furthermore, we continued to experience growth momentum in our pipeline, including receiving 12 approvals across Takeda’s key markets.

"With FY2021 anticipated to serve as a critical inflection year, we remain focused on leveraging our expected topline growth to ramp up our R&D investment and further fuel our transformative pipeline. We are well-positioned to reach our goal of mid-single-digit revenue growth over the next decade amounting to JPY5 trillion ($47 billion) by FY2030.1

"As we celebrate Takeda’s 240th anniversary next month, I am extremely proud of our progress and confident in our outlook for the future. We will advance on our growth trajectory, maximize value creation for all of our stakeholders, and continue to position the company for long-term success."

FINANCIAL AND BUSINESS HIGHLIGHTS

Results for FY2020 Ended March 31, 2021

(a) Further information on certain of Takeda’s Non-IFRS measures is posted on Takeda’s investor relations website at View Source
(b) Underlying growth compares two periods (quarters or years) of financial results under a common basis and is used by management to assess the business. These financial results are calculated on a constant currency basis and excluding the impact of divestitures and other amounts that are unusual, non-recurring items or unrelated to our ongoing operations.
(c) Core Operating Profit represents net profit adjusted to exclude income tax expenses, the share of profit or loss of investments accounted for using the equity method, finance expenses and income, other operating expenses and income, amortization and impairment losses on acquired intangible assets and other items unrelated to Takeda’s core operations, such as purchase accounting effects and transaction related costs.
(d) Free Cash Flow represents cash flows from operating activities, excluding acquisition of plant, property and equipment, intangible assets and investments, and any other cash that is not available to Takeda’s immediate or general business use, and including proceeds from sales of plant, property and equipment, as further adjusted to exclude the acquisition of intangible assets and the acquisition of investments, and to include the proceeds from sales of property, plant, sales and redemption of investments and businesses, net of cash and cash equivalents divested.

FY2020 RESULTS DEMONSTRATE TAKEDA’S RESILIENT PORTFOLIO

Reported revenueat JPY 3,197.8 billion (~$28.9B)2, declined by 2.8% impacted primarily by foreign exchange and divestitures. Underlying revenue growth in FY2020 was +2.2% driven by the growth of Takeda’s 14 global brands, up 16% year-on-year.
Takeda delivered reported operating profit of JPY 509.3 billion (~$4.6B)2, which grew 407.2% with gains from non-core asset sales and acquisition-related expenses. Core operating profit, which adjusts for purchase price accounting ("PPA") and non-recurring items (including gains on sales of assets), increased year-on-year to JPY 967.9 billion (~$8.8B)2. The core operating profit margin was 30.3%. Underlying core operating profit, which further adjusts for the impact of foreign exchange and divestitures, grew 13% year-on-year. The underlying core operating profit margin was 30.2%.
Takeda’s reported net profit was JPY 376 billion, a 749.9% increase compared with the same period in the prior year.
Operating cash flow increased by 50.9% to JPY 1,010.9 billion. There was an increase in other financial liabilities of JPY 175.5 billion primarily attributable to an increase of deposits restricted to certain vaccines operations.
Free cash flow, which adjusts out deposits restricted to certain vaccine operations and reflects capital expenditures and proceeds from asset sales, was JPY 1,237.8 billion (~$11.2B)2. This represented an increase of 27.9% versus the prior year, comfortably covering the full year dividend, debt repayment and interest. Robust cash flow enabled further de-leveraging in Q4.
The overall impact of the global spread of COVID-19 on Takeda’s consolidated financial results for the twelve-month period ended March 31, 2021, was not material, with several offsetting factors. There were adverse effects due to COVID-19 observed in certain therapeutic areas, especially in Neuroscience when stay-at-home restrictions reduced patient visits to medical care providers. This trend has fluctuated throughout the twelve-month period. These adverse effects have been partially offset by benefits from prescribing trends during the pandemic, such as an expansion of certain products with a more convenient administration profile. Regarding operating expenses, voluntary suspension of certain business activities such as business travel and events in response to COVID-19 led to lower spending. As a result of these factors, the impact on Takeda’s profit was immaterial.

For the latest Takeda communications regarding COVID-19, please click here to visit the COVID-19 Information Center on Takeda’s website.

COMMERCIAL UPDATES ACROSS FIVE KEY BUSINESS AREAS

Takeda’s five key business areas — Gastroenterology, Rare Diseases, Plasma-Derived Therapies, Oncology and Neuroscience — with JPY 2,623.7 billion of reported revenue representing approximately 82% of total FY2020 revenues – delivered year-on-year underlying revenue growth of 4.7%. Takeda’s 14 global brands, with reported revenue of JPY 1,215.3 billion (~$11.0B) 2 in aggregate, delivered a 16% increase in FY2020 underlying revenue growth compared to a year before.

Gastroenterology

Gastroenterology with JPY 777.8 billion in reported revenue represented 24% of sales, with underlying revenue growth of 14%. This was spearheaded by continued exceptional growth through expanded first line share of gut selective ENTYVIO in the U.S., EU and Japan.

Rare Diseases

Rare Diseases with JPY 591.7 billion in reported revenue represented 19% of sales, with underlying revenue declining 2% driven by a decline in rare hematology that was in line with expectations. The hereditary angioedema (HAE) portfolio saw 10% underlying revenue growth, driven by continued excellent performance from TAKHZYRO, which continues to expand the hereditary angioedema prophylaxis market, as well as launching into additional geographies. Rare Metabolic increased 2% on an underlying basis but excluding NATPARA the portfolio saw 8% underlying growth.

PDT Immunology

PDT Immunology with JPY 420.4 billion in reported revenue represented 13% of sales, with underlying revenue growth of 10% driven by strong Gammagard-Liquid demand in the U.S. and subcutaneous IG worldwide. Albumin underlying revenue declined 13% in FY2020, mostly due to H2 sales impact caused by temporary interruption in submitting batches of Albumin Glass for release in China, and in some part due to phasing and supply dynamics in China in FY2019. COVID-19 has impacted plasma collections industry-wide, but operational excellence, implementation of digital initiatives and ongoing center expansion helped limit plasma collection volume decline to only -11%.

Oncology

Oncology with JPY 416.5 billion in reported revenue represented 13% of sales, with underlying revenue growth of 1% driven by NINLARO, ADCETRIS and ALUNBRIG. Takeda’s strong oncology portfolio continues to expand indications as growth brands offset the decline of older products in the portfolio.

Neuroscience

Neuroscience with JPY 417.3 billion in reported revenue represented 13% of sales, declining 2% on an underlying basis. The portfolio experienced a slowdown in momentum attributable to COVID-19 stay-at-home restrictions that reduced patient visits and diagnoses. A recovery of prescribing trends has been noted, but new patient starts are not yet back to pre-COVID levels.

COST SAVINGS AND DIVESTITURES

Synergy deliverables and operational efficiencies supported margin performance, as Takeda delivered an underlying core operating profit margin of 30.2%. Takeda is also deleveraging rapidly, with a net debt/adjusted EBITDA ratio of 3.2x at the end of Q4, down from 3.8x in March 2020. Net debt has decreased by JPY 1,668.5 billion in two years since March 31, 2019 (the Shire Acquisition closed on January 8, 2019), and Takeda is on course to meet its medium-term deleveraging goal of 2x (low-twos) within FY2021-FY2023.

Takeda exceeded its $10B non-core asset divestiture target and has announced 12 deals since January 2019 to date for a total aggregate value of up to ~$12.9 billion3, most recently including:

The completion of the previously announced sale of a portfolio of four type 2 diabetes products to Teijin Pharma Limited for a total value of JPY 133.0 billion.4 (Press Release)
The completion of the previously announced sale of a portfolio of approximately 130 select over the counter and prescription products, and two manufacturing sites to Orifarm for a total value of up to $670 million USD. (Press Release)
The completion of the previously announced sale of Takeda Consumer Healthcare Company Limited to Oscar A-Co KK, a company controlled by funds managed by The Blackstone Group Inc. and its affiliates, for a total value of JPY 227.7 billion.5 (Press Release)
Takeda has also exceeded its original $700 million target for incremental cash from sales of real estate and marketable securities in FY2020, receiving a total of ~$1.4B.

PIPELINE UPDATE: FY2021 – ANTICIPATED TO BE AN INFLECTION YEAR WITH FIVE TO SIX WAVE 1 NMES SUBMITTED AND UNDER REGULATORY REVIEW BY THE FDA WITH THE POTENTIAL FOR FOUR APPROVALS

Takeda’s world-class R&D engine is fueled by leveraging internal research capabilities and actively engaging with innovative ecosystems around the world. This efficient R&D model has enabled us to focus on more targeted patient populations where there is potential for greater therapeutic benefit, smaller and less costly development programs, and faster tracks to registration with enhanced patent protection and marketing rights. We plan to increase our R&D investment to 522 billion JPY to further advance our 40+ prioritized NMEs and new partnerships, as well as building additional capabilities in oncology, clinical trial initiation and data and digital sciences. In FY2020, we obtained 12 global and regional brand approvals with the U.S., EU, China and Japan, demonstrating our drive to develop best-in-class therapies for patients with high unmet needs around the world. FY2021 is expected to be an inflection year for Takeda as we anticipate up to six regulatory submissions by year-end FY2021, with the potential for four approvals.

Takeda’s pipeline portfolio has the potential to contribute significantly to its growth over the next decade, with recent highlights including:

TAK-003:Takeda’s tetravalent dengue vaccine candidate, has completed its first regulatory submissions with possible approval in the EU and some endemic countries in FY2021. (Press release)
Mobocertinib (TAK-788): A potential new oral standard of care for adult patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small cell lung cancer (mNSCLC), has completed its New Drug Application (NDA) submission with a potential approval in FY2021. (Press release)
Maribavir (TAK-620): A robust primary analysis from the Phase 3 trial showed significantly more patients achieved cytomegalovirus (CMV) clearance versus conventional therapies in transplant recipients with CMV infection. Met key secondary endpoints, maintaining superior CMV viremia clearance, on track for NDA submission. Takeda continues to investigate Maribavir in the ongoing 302 Phase 3 study for First-Line Treatment of CMV in Hematopoietic Cell Transplant Recipients. (Press release)
Soticlestat (TAK-935): Takeda recently re-acquired global rights from Ovid Therapeutics to develop and commercialize this potential first-in-class therapy, with a novel mechanism of action for the treatment of developmental and epileptic encephalopathies. We intend to initiate Phase 3 studies of soticlestat in children and young adults with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in FY2021. If successful, it has the potential to bring new treatment options that provide greater seizure control, tolerability and function to DS and LGS patients around the world. (An Inflection Year for Our Wave 1 Pipeline)
Orexin (TAK-994): Potentially the first therapy to treat orexin deficiency, Takeda plans to move this asset into registrational trials, first in narcolepsy type 1 (NT1) — a rare, underdiagnosed and undertreated condition caused by an orexin deficiency which disrupts the sleep awake cycles, with narcolepsy type 2 (NT2) and idiopathic hypersomnia (IH) to follow as potential additional indications. (An Inflection Year for Our Wave 1 Pipeline)
TAK-186: A conditionally active T-cell engager, first in its class to enter the clinic with recent phase 1/2 study initiation in EGFR-expressing solid tumors. TAK-186 is one of the latest additions to our pipeline through the acquisition of Maverick Therapeutics. In addition to TAK-186, Takeda also obtained Maverick’s T-cell engager COBRA platform and TAK-280 which is expected to enter the clinic in the second half of FY2021 for the treatment of patients with B7H3-expressing solid tumors. (Press release)
KEY CORPORATE INITIATIVES

Several recent examples of Takeda’s corporate achievements in FY2020 demonstrate Takeda’s progress toward its purpose of "better health for people, brighter future for the world":

Patients:

Launched R&D Center for Health Equity and Patient Affairs to identify and address health inequities.
Developed the Health Outcomes Observatory (H20) project, which brings together diverse public and private partners to amplify the patient voice in Europe.
Awarded the 2021 Facility of the Year Awards (FOYA) by the International Society for Pharmaceutical Engineering (ISPE) in two categories. Takeda’s new solid pharmaceutical packaging building in Hikari, Japan, was recognized with the 2021 "Process Intelligence and Innovation" category award and the end-to-end high potent drug facility in Grange Castle, Ireland, was selected as "Facility Integration" category winner.
Earned an industry-leading position within the 2021 Access to Medicine (AtM) Index where the company ranked sixth overall and led the pharmaceutical industry in Governance of Access.

People:

Launched our first Global DE&I Council, led by members of the Takeda Executive Team, to further embed DE&I into our culture.
Achieved global Top Employer certification for fourth consecutive year and was named as a Top Employer in four regions and 38 countries.
Preparing for post-pandemic ways of working with new hybrid working models that foster a flexible working culture, aligned to local business needs, that optimize employee engagement.
Planet:

Achieved carbon neutrality in the value chain for fiscal year 2019.
Named to Corporate Knights Global 100 Most Sustainable Corporations in the World (Global 100) for the sixth consecutive year.
Governance:

Takeda recently announced candidates for its Board of Directors that will be proposed at the 145th Ordinary General Meeting of Shareholders to be held on June 29, 2021. As part of its commitment to exercising strong corporate governance practices, Takeda decided that all members of the Audit and Supervisory Committee will be external directors (as defined under the rules of the Tokyo Stock Exchange) to further enhance the independence of the Committee. This change will promote the long-term interests of shareholders and all its stakeholders as well as strengthening its Board of Directors and management accountability. (Press release)
COVID-19 UPDATE

Guided by its values, Takeda’s response to COVID-19 has focused on protecting the health and safety of employees, striving to ensure its medicines are available to patients who rely on them and playing a part to reduce transmission and support the communities where its employees live and work.

While the results of our CoVIg-19 Plasma Alliance clinical trial were not favorable, the effort strengthened relationships within and outside the industry, enabled a renewed perspective toward pragmatic regulation based on scientific evidence and need, and provided a well-defined, legally compliant framework for future collaborative opportunities to address urgent public health needs. Takeda has also undertaken several efforts to help the world respond to COVID-19, and our most recent accomplishments include:

Takeda is making two COVID-19 vaccines available in Japan, by manufacturing Novavax’ recombinant vaccine candidate and distributing Moderna’s mRNA vaccine candidate, with the support of the Ministry of Health, Labour and Welfare and the Japan Agency for Medical Research and Development (AMED). Pending regulatory approval, Takeda intends to start distributing TAK-919 (Moderna) in the first half of 2021 and aims to start distributing TAK-019 (Novavax) in late 2021 or early 2022. (Press Release)
Takeda and IDT Biologika GmbH (IDT) have a mutual agreement to support manufacturing of Johnson & Johnson’s COVID-19 vaccine for three months utilizing capacity previously reserved for Takeda’s dengue vaccine candidate. (Press Release)

FY2021 GUIDANCE: Growth Momentum Expected to Continue

Takeda has solid growth momentum heading into FY2021 and expects underlying revenue growth to accelerate to "mid-single-digit" driven by continued momentum of Takeda’s 14 global brands.

Reported revenue is forecast to be 3,370 billion JPY, a year-on-year increase of 172.2 billion JPY or +5.4% from FY2020, with underlying revenue momentum and a one-time gain from the sale of diabetes portfolio in Japan fully offsetting impacts from divestitures completed in FY2020.

Underlying Core Operating Profit and Underlying Core EPS are expected to also grow at "mid-single-digit", reflecting revenue growth and continued cost efficiencies, whilst also incorporating a significant increase in R&D expenses to support Takeda’s innovative pipeline.

Reported Operating Profit is expected to be 488 billion JPY, a decrease of 21.3 billion JPY, or -4.2%, impacted by a significant increase in R&D expenses as well as lower one-time gains on asset sales. Core Operating Profit is expected to decrease by 37.9 billion JPY, or -3.9%, to 930 billion JPY, reflecting a significant increase in R&D expenses. Reported net profit for the year is expected to be 250 billion JPY, a decrease of 126 billion JPY, or -33.5%, reflecting the impacts on reported operating profit as well as an expected increase in the effective tax rate.

Key Assumptions in FY2021 Forecast

Company guidance reflects management’s expectations for continued business momentum across Takeda’s five key business areas, underlying revenue growth of its 14 global brands, and accelerated realization of cost synergies.

FY2021 guidance also reflects the following key assumptions: (i) The gain on sale of a diabetes portfolio in Japan is booked as revenue (JPY 133 billion), and adjusted out of Core Operating Profit for FY2021; (ii) Takeda expects at least one 505(b)2 competitor for subcutaneous VELCADE to launch in the U.S. around mid FY2021; (iii) Takeda does not expect to restart sales of NATPARA in the U.S. market in FY2021; (iv) FY2021 guidance does not include the impact of any potential further divestitures beyond what has already been disclosed by Takeda.

Based on currently available information, Takeda believes its financial results for FY2021 will not be materially affected by COVID-19 and, accordingly, Takeda’s FY2021 forecast reflects this belief. However, the situation surrounding COVID-19 remains highly fluid, and future COVID-19-related developments in FY2021, including new or additional COVID-19 outbreaks and additional or extended lockdowns, shelter-in-place orders or other government action in major markets, could result in further or more serious disruptions to Takeda’s business, such as slowdowns in demand for Takeda’s products, supply chain related issues or significant delays in its clinical trial programs. These events, if they occur, could result in additional impacts on Takeda’s business, results of operations or financial condition, as well as resulting in significant deviations from Takeda’s FY2021 forecast.

For more details on Takeda’s FY2020 results and other financial information, please visit: View Source

Further Information

Takeda will share details regarding its commercial strategies in oncology and finance at its upcoming Oncology Strategic Update Call in June 2021 and Finance Strategy Day in June/July 2021, respectively (dates to be confirmed). Additionally, Takeda will share details regarding COVID-19 efforts, the current state of the business, and the short- and long-term outlook of the Company at the Annual General Meeting of Shareholders​ on June 29, 2021.

On May 11, 2021 Ultimovacs ASA ("Ultimovacs", ticker ULTI), a pharmaceutical company developing novel immunotherapies against cancer, reported its first quarter 2021 results today (Press release, Ultimovacs, MAY 11, 2021, View Source [SID1234579701]). A presentation by the Company’s management team will take place today on a webcast at 09:00 CEST. The presentation can be followed as a live webcast, which will also be available on our website.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Highlights for the first quarter of 2021:

In the INITIUM trial, 40 patients have been enrolled as per the reporting date as compared to 24 patients reported in the previous quarterly report.
In the NIPU trial, 29 patients have been enrolled as per the reporting date compared to 18 patients reported in the previous quarterly report.
As a natural consequence of the COVID-19 pandemic, the activation of hospitals is progressing slower than initially planned in both the INITIUM and NIPU trials. The Company continues to implement activities to minimize the impact on patient recruitment. The effect of the pandemic on the biotech industry and the general ability to conduct clinical trials is still uncertain and dependent on the speed of return to a more normal situation.
Ultimovacs provided details on the DOVACC trial in January 2021. Ultimovacs will participate in this randomized Phase II collaboration study, together with the Nordic Society of Gynaecological Oncology – Clinical Trial Unit (NSGO-CTU), the European Network of Gynaecological Oncological Trial Groups (ENGOT) and AstraZeneca, to evaluate Ultimovacs’ proprietary universal cancer vaccine, UV1, in combination with AstraZeneca’s durvalumab and olaparib in patients with relapsed ovarian cancer. The trial will include 184 patients in approximately 10 European countries at more than 40 sites. (Announced in Q4-20 report).
Preparations are ongoing for the initiation of the DOVACC and FOCUS trials, with the first patients expected to be included in both trials around mid-year 2021.
On 18 February 2021, Ultimovacs started clinical evaluation of the novel TET-Platform, with the treatment of the first patient in the Phase I TENDU study investigating a prostate cancer-specific therapeutic vaccine. The first cohort of three patients has been enrolled as per the reporting date.
On 28 April 2021, Ultimovacs announced that an abstract on the Company’s Phase I trial evaluating its universal cancer vaccine, UV1, in combination with the checkpoint inhibitor pembrolizumab in patients with metastatic malignant melanoma has been accepted for a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting to be held virtually from June 4 to 8, 2021. (post period event).
Total operating expenses amounted to MNOK 31.2 in Q1-21.
Cash flow from operations was MNOK -29.5 in Q1-21. Total cash and cash equivalents amounted to MNOK 409.3 as per 31 March 2021.